PBGENE-DMD is an investigational first-in-class in vivo gene-editing therapy for Duchenne muscular dystrophy in patients with dystrophin mutations between exons 45-55. It uses a gene-excision approach intended as a one-time treatment to restore near full-length dystrophin production. FDA provided a Study May Proceed notification in February 2026, enabling Phase 1/2 trial site activation.
Precision BioSciences receives FDA Fast Track designation for PBGENE-DMD, a gene editing therapy for Duchenne muscular dystrophy designed to treat patients with mutations in exons 45-55.
Precision BioSciences announced FDA Fast Track designation for PBGENE-DMD, a first-in-class gene editing therapy for Duchenne muscular dystrophy targeting mutations in exons 45-55, representing up to 60% of DMD patients.
